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Ganaxolone for Epilepsy

Phase 2
Waitlist Available
Research Sponsored by Marinus Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant
Failure to control seizures despite 2 or more anti-seizure medications
Timeline
Screening 3 weeks
Treatment Varies
Follow Up end of the double-blind 17 week treatment period
Awards & highlights

Study Summary

This study is evaluating whether ganaxolone is safe and effective for treating seizures in children and young adults with PCDH19 gene mutation.

Eligible Conditions
  • Epilepsy

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~end of the double-blind 17 week treatment period
This trial's timeline: 3 weeks for screening, Varies for treatment, and end of the double-blind 17 week treatment period for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
Secondary outcome measures
50% Primary Seizure Reduction
Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)

Side effects data

From 2014 Phase 2 trial • 112 Patients • NCT01339689
19%
Somnolence
14%
Headache
8%
Fatigue
8%
Sedation
7%
Dizziness
7%
Nightmare
7%
Vision blurred
7%
Myalgia
5%
Confusional state
5%
Dysarthria
3%
Vertigo
3%
Pain in extremity
3%
Gastrointestinal disorder
3%
Tremor
3%
Neck pain
3%
Nasopharyngitis
3%
Increased appetite
3%
Ataxia
3%
Balance disorder
3%
Cough
3%
Insomnia
3%
Diarrhoea
3%
Lethargy
2%
Protein urine present
2%
Rhinorrhoea
2%
Urinary tract infection
2%
Confusion
2%
Upper respiratory tract infection
2%
Arthralgia
2%
Dry mouth
2%
Road traffic accident
2%
Tandem galt test abnormal
2%
Pulmonary congestion
2%
Balance Issues
2%
Splenomegaly
2%
Vomiting
2%
Chest pain
2%
Decreased appetite
2%
Abnormal dreams
2%
Nausea
2%
Chest discomfort
2%
Feeling drunk
2%
Irritability
2%
Pharyngitis streptococcal
2%
Blood urine present
2%
Weight increased
2%
Back pain
2%
Muscle spasms
2%
Depressed level of consciousness
2%
Disturbance in attention
2%
Motor dysfunction
2%
Anger
2%
Depressed
2%
Euphoric mood
2%
Initial insomnia
2%
Psychomotor retardation
2%
Sleep disorder
2%
Upper airway resistance syndrome
2%
Anorgasmia
2%
Suicidal ideation
2%
Haematuria
2%
Suicide of companion
2%
Product used for unknown indication
2%
Dry Eye
2%
Abdominal discomfort
2%
Alanine aminotransferase increased
2%
Liver function test abnormal
2%
Tic
2%
Migraine
2%
Suicidal Ideation
2%
Fever
2%
Sinus congestion
2%
Rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ganaxolone
Placebo

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: GanaxoloneExperimental Treatment1 Intervention
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Group II: PlaceboPlacebo Group1 Intervention
placebo suspension 3x's /day for 17 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxolone
FDA approved

Find a Location

Who is running the clinical trial?

Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,934 Total Patients Enrolled
6 Trials studying Epilepsy
577 Patients Enrolled for Epilepsy
Maciej Gasior, M.D., Ph.DStudy DirectorMarinus Pharmaceuticals, Inc.
Paula Bokesk, M.D., FAAPStudy DirectorMarinus Pharmaceuticals, Inc.

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~5 spots leftby Mar 2025